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EP3678710A4 - Adn à extrémité fermée (cedna) modifié - Google Patents

Adn à extrémité fermée (cedna) modifié Download PDF

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Publication number
EP3678710A4
EP3678710A4 EP18854941.4A EP18854941A EP3678710A4 EP 3678710 A4 EP3678710 A4 EP 3678710A4 EP 18854941 A EP18854941 A EP 18854941A EP 3678710 A4 EP3678710 A4 EP 3678710A4
Authority
EP
European Patent Office
Prior art keywords
cedna
ended dna
modified closed
modified
closed
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
EP18854941.4A
Other languages
German (de)
English (en)
Other versions
EP3678710A1 (fr
Inventor
Robert Michael Kotin
Ozan ALKAN
Annaliese JONES
Douglas Anthony KERR
Ara Karl MALAKIAN
Matthew John Simmons
Teresa L. WRIGHT
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Generation Bio Co
Original Assignee
Generation Bio Co
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Generation Bio Co filed Critical Generation Bio Co
Publication of EP3678710A1 publication Critical patent/EP3678710A1/fr
Publication of EP3678710A4 publication Critical patent/EP3678710A4/fr
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/66—General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
    • C12N2710/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2710/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/60—Vectors comprising a special translation-regulating system from viruses

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
EP18854941.4A 2017-09-08 2018-09-07 Adn à extrémité fermée (cedna) modifié Pending EP3678710A4 (fr)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762556331P 2017-09-08 2017-09-08
US201762556319P 2017-09-08 2017-09-08
US201762556329P 2017-09-08 2017-09-08
US201762556335P 2017-09-08 2017-09-08
US201762556324P 2017-09-08 2017-09-08
US201762556281P 2017-09-08 2017-09-08
PCT/US2018/049996 WO2019051255A1 (fr) 2017-09-08 2018-09-07 Adn à extrémité fermée (cedna) modifié

Publications (2)

Publication Number Publication Date
EP3678710A1 EP3678710A1 (fr) 2020-07-15
EP3678710A4 true EP3678710A4 (fr) 2021-06-09

Family

ID=65635235

Family Applications (1)

Application Number Title Priority Date Filing Date
EP18854941.4A Pending EP3678710A4 (fr) 2017-09-08 2018-09-07 Adn à extrémité fermée (cedna) modifié

Country Status (15)

Country Link
US (1) US20200283794A1 (fr)
EP (1) EP3678710A4 (fr)
JP (3) JP2020532981A (fr)
KR (1) KR20200051011A (fr)
CN (1) CN111132699A (fr)
AU (1) AU2018327348A1 (fr)
BR (1) BR112020004151A2 (fr)
CA (1) CA3075168A1 (fr)
IL (1) IL272797A (fr)
MA (1) MA50100A (fr)
MX (1) MX2020002500A (fr)
PH (1) PH12020500465A1 (fr)
SG (1) SG11202000698SA (fr)
UA (1) UA129010C2 (fr)
WO (1) WO2019051255A1 (fr)

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CA2935960C (fr) 2014-01-08 2023-01-10 Bart Lipkens Dispositif d'acoustophorese avec double chambre acoustophoretique
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
AU2018313921B2 (en) 2017-08-09 2024-12-19 Bioverativ Therapeutics Inc. Nucleic acid molecules and uses thereof
CN111527200A (zh) * 2017-12-06 2020-08-11 世代生物公司 使用修饰的封闭端DNA(ceDNA)的基因编辑
WO2019118921A1 (fr) 2017-12-14 2019-06-20 Flodesign Sonics, Inc. Circuit d'excitation et circuit de commande de transducteur acoustique
WO2019161059A1 (fr) * 2018-02-14 2019-08-22 Generation Bio Co. Vecteurs d'adn non viraux et utilisations associées pour la production d'anticorps et de protéines de fusion
MA54188A (fr) * 2018-11-09 2021-09-15 Generation Bio Co Adn à extrémité fermée modifié (cedna) comprenant des répétitions terminales inversées modifiées symétriques
GB201905651D0 (en) * 2019-04-24 2019-06-05 Lightbio Ltd Nucleic acid constructs and methods for their manufacture
AU2020314865A1 (en) * 2019-07-17 2021-12-23 Generation Bio Co. Compositions and production of nicked closed-ended DNA vectors
AU2020315444A1 (en) * 2019-07-17 2021-12-23 Generation Bio Co. Synthetic production of single-stranded adeno associated viral DNA vectors
JP2022546597A (ja) * 2019-09-06 2022-11-04 ジェネレーション バイオ カンパニー 閉端ｄｎａおよび切断可能脂質を含む脂質ナノ粒子組成物ならびにそれらの使用方法
EP4031168A4 (fr) * 2019-09-18 2023-10-11 Intergalactic Therapeutics, Inc. Vecteurs d'adn synthétiques et procédés d'utilisation
WO2021072031A1 (fr) * 2019-10-11 2021-04-15 Insideoutbio, Inc. Procédés et compositions pour la fabrication et l'utilisation d'agents thérapeutiques codés par de l'adn circulaire dans des troubles génétiques et d'autres maladies
WO2021169167A1 (fr) * 2020-02-29 2021-09-02 Nanjing GenScript Biotech Co., Ltd. Méthode de traitement d'infections à coronavirus
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
CN116234917A (zh) 2020-07-27 2023-06-06 安杰瑞姆生物科学公司 Dna分子组合物及其制备方法和使用方法
BR112023002395A2 (pt) 2020-08-23 2023-03-21 Bioverativ Therapeutics Inc Sistema de baculovírus modificado para produção aprimorada de dna com extremidades fechadas (cedna)
IL301196A (en) * 2020-09-16 2023-05-01 Generation Bio Co Closed-ended dna vectors and uses thereof for expressing phenylalanine hydroxylase (pah)
GB202014751D0 (en) * 2020-09-18 2020-11-04 Lightbio Ltd Targeting vector
US20240209393A1 (en) * 2021-04-26 2024-06-27 University Of Florida Research Foundation, Incorporated Synthetic aav vectors for repeated delivery of therapeutic genes
WO2022232289A1 (fr) * 2021-04-27 2022-11-03 Generation Bio Co. Vecteurs d'adn non viraux exprimant des anticorps thérapeutiques et leurs utilisations
EP4329884A1 (fr) * 2021-04-27 2024-03-06 Generation Bio Co. Vecteurs d'adn non viraux exprimant des anticorps anti-coronavirus et leurs utilisations
EP4333906A4 (fr) * 2021-05-07 2025-04-02 Generation Bio Co Compositions de vecteurs d'adn non viraux lyophilisées et leurs utilisations
CN117729934A (zh) * 2021-05-07 2024-03-19 世代生物公司 用于疫苗递送的非病毒dna载体
JP2024538168A (ja) * 2021-10-18 2024-10-18 フラッグシップ パイオニアリング イノベーションズ セブン，エルエルシー Ｄｎａ組成物及び関連方法
CN119072330A (zh) 2022-03-14 2024-12-03 世代生物公司 异源初免加强疫苗组合物和使用方法
CN117802161A (zh) * 2022-06-30 2024-04-02 苏州吉恒基因科技有限公司 精准重组腺相关病毒载体及其用途
WO2024040222A1 (fr) * 2022-08-19 2024-02-22 Generation Bio Co. Adn à extrémités fermées clivable (adnce) et ses procédés d'utilisation
WO2024182707A1 (fr) 2023-03-02 2024-09-06 Krystal Biotech, Inc. Interleukine-2 et interleukine-12 pour le traitement du cancer
US11891617B1 (en) * 2023-03-27 2024-02-06 Genecraft Inc. Modified complex platform of adeno-associated virus with improved rate of expression of loaded genes and reduced genotoxicity
US11767541B1 (en) * 2023-03-27 2023-09-26 Genecraft, Inc. Adeno-associated virus complex with improved expression of RUNX3 gene and uses for preventing or treating KRAS mutated lung cancer
US11993783B1 (en) * 2023-03-27 2024-05-28 Genecraft Inc. Nucleic acid molecule comprising asymmetrically modified ITR for improving expression rate of inserted gene, and use thereof

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Also Published As

Publication number Publication date
CA3075168A1 (fr) 2019-03-14
RU2020109904A (ru) 2021-10-08
BR112020004151A2 (pt) 2020-09-08
JP2025010253A (ja) 2025-01-20
MA50100A (fr) 2020-07-15
WO2019051255A1 (fr) 2019-03-14
JP2022190081A (ja) 2022-12-22
CN111132699A (zh) 2020-05-08
MX2020002500A (es) 2020-09-17
NZ761178A (en) 2024-05-31
UA129010C2 (uk) 2024-12-25
JP2020532981A (ja) 2020-11-19
PH12020500465A1 (en) 2021-01-25
AU2018327348A1 (en) 2020-02-20
IL272797A (en) 2020-04-30
KR20200051011A (ko) 2020-05-12
SG11202000698SA (en) 2020-03-30
EP3678710A1 (fr) 2020-07-15
US20200283794A1 (en) 2020-09-10

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